Gene Therapy on the Verge of Taking its Next Evolutionary Step

Brand Insights - Thought Leadership | Paid Program

The shift from (ultra) rare to prevalent diseases calls for new vector manufacturing technologies

Nicole, with this year’s cell & gene conference season in full swing, can you highlight a few key messages?

The cell & gene therapy sector is really gaining momentum. We see a lot of progress in clinical pipelines and many new approvals. According to the latest report from the Alliance for Regenerative Medicine, there are nine regulatory decisions in gene therapy expected from the FDA alone within the next year, plus seven in Europe. And from our discussions with Pharma, we know that there is enormous clinical and preclinical activity in the sector.

Speaking of gene therapy: You have been active in this industry for quite a while now. How do you see it evolving and have you seen anything that marks a notable change?

Looking at vector types, there has been a clear shift towards AAV for gene therapy applications. For in vivo gene therapy, AAV is now the predominant vector type. Critically, we also have seen a clear shift from rare and ultra-rare to prevalent diseases. This means larger trials and higher patient numbers, posing new challenges for the manufacturing process. Gene therapy is really on the verge of taking its next evolutionary step.

What does that mean in terms of new needs, specifically in vector manufacturing?

With higher amounts of vectors required, manufacturing processes need to become more robust, reliable and, foremost, scalable. Platform technologies are needed to enable this. That’s why we at CEVEC - as an enabling technology provider - are getting a lot of attention right now.

So, the classical standard for AAV, triple plasmid transient transfection, does not do the job?

Correct. Transient transfection of cells using plasmids works well in smaller volumes and academic settings. But it lacks scalability, reproducibility and efficiency, all needed for gene therapies at larger scales.

What kind of production technology is the industry looking for?

The future of scalable vector manufacturing is stable producer cell lines. We saw this 20 years ago with monoclonal antibodies. Therefore, CEVEC has developed ELEVECTA® - the first and only AAV manufacturing technology based on inducible stable producer cell lines. With ELEVECTA®, all components for AAV production are stably integrated into the genome of a suspension cell line. All you need for manufacturing is the producer cell and the standardized production process in stirred tank bioreactors. No helper virus, no plasmids.

Another industry that has gained momentum during the pandemic is the vaccine industry.Does the manufacturing of vector-based vaccines face the same challenges?

Generally speaking, yes. However, vaccines have to fulfill highest safety standards. Especially for adenoviral vectors, the absence of replication-competent adenovirus is particularly important. CEVEC’s CAP® Ad platform has been specifically designed to ensure safe adenoviral vector production.

Your company gets a lot of attention in the industry. Can you name clients that are using your technologies, specifically in the viral vector space?

Our aim is to make our leading technologies widely available and to be a reliable and competent partner to the Pharma industry. This approach has been well received. Most of our clients are US-based, such as Biogen, but Europe is catching up, as you can see from our collaborations with Roche and UCB.

So, what is next? For you as a company and, in general, for the industry?

Clearly, expanding capacity to meet the rising market demand is a top priority. We are looking to team up with strong partners that complement our expertise and capabilities.The goal of partnering is to further strengthen our leading market position and, with that, to advance the gene therapy industry as a whole.